RARECast

516 Episodes

1. Targeting an Immune Switch in Rare Diseases

   Published: 4/1/2020
2. Targeting the Regulatory Genome to Treat Rare Blood Diseases

   Published: 3/25/2020
3. A Reborn Forma Therapeutics Focuses on Rare Blood Diseases and Cancer

   Published: 3/18/2020
4. Targeting Genetic Diseases at Their Root Cause

   Published: 3/11/2020
5. Targeting Hormone Dysregulation in Prader-Willi Syndrome

   Published: 3/4/2020
6. Rare Disease Advocates Take to the Hill

   Published: 2/25/2020
7. Targeting a Common Link in Otherwise Different Rare Diseases

   Published: 2/19/2020
8. Bringing Clinical Trials to Patients

   Published: 2/12/2020
9. Crowdsourcing Therapeutic Approaches for an Ultra-Rare Disease

   Published: 2/5/2020
10. Delivering Oligonucleotide Therapies to Any Cell Type

    Published: 1/29/2020
11. Protalix Looks Beyond Biobetters

    Published: 1/22/2020
12. Driving N-of-1 Therapies for Ultra-Rare Disease Patients

    Published: 1/15/2020
13. Cutting the Time and Cost of Drug Discovery with MicroOrgans

    Published: 1/8/2020
14. Acquisition Combines Two Pipelines for Rare Skin Diseases

    Published: 1/2/2020
15. Programming Cells to Produce Neuroprotective Factors to Treat ALS

    Published: 12/26/2019
16. Origin's Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition

    Published: 12/18/2019
17. Developing a Common Language for Rare Diseases

    Published: 12/11/2019
18. Why Payers Lag Drug Developers and Regulators in Embracing Real-World Evidence

    Published: 12/4/2019
19. How a Brother's Love Is Forging a Path for Customized Therapies for Rare Disease Patients

    Published: 11/27/2019
20. Addressing the Barriers to Accessing Experimental Therapies

    Published: 11/20/2019